NCT03346434|CompletedPhase 2ResultsDMCFDA Drug

Safety, Pharmacokinetics and Efficacy of Dupilumab in Patients ≥6 Months to <6 Years With Moderate-to-Severe Atopic Dermatitis (Liberty AD PRESCHOOL)

Liberty AD

A Phase 2/3 Study Investigating the Pharmacokinetics, Safety, and Efficacy of Dupilumab in Patients Aged ≥6 Months to <6 Years With Moderate-to-Severe Atopic Dermatitis

Regeneron Pharmaceuticals ClinicalTrials.gov

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2 other identifiers

R668-AD-15392016-000955-28

Study Type

interventional

Target

202

Locations

4 countries

Sites

Timeline

RegisteredNov 2017

StartedNov 2017

CompletionJul 2021

Brief Summary

This study is a 2-part (parts A and B) phase 2/3 study to evaluate the safety, pharmacokinetics (PK) and efficacy of dupilumab in participants 6 months to less than 6 years of age with moderate-to-severe atopic dermatitis (AD).

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

202

participants targeted

Target at P75+ for phase_2

Timeline

Completed

Started Nov 2017

Typical duration for phase_2

Geographic Reach

4 countries

48 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

3.6 years study duration

First Submitted

Initial submission to the registry

February 6, 2017

Completed

9 months until next milestone

First Posted

Study publicly available on registry

November 17, 2017

Completed

13 days until next milestone

Study Start

First participant enrolled

November 30, 2017

Completed

3.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 8, 2021

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 8, 2021

Completed

1.1 years until next milestone

Results Posted

Study results publicly available

July 28, 2022

Completed

Last Updated

July 28, 2022

Status Verified

July 1, 2022

Enrollment Period

3.6 years

First QC Date

February 6, 2017

Results QC Date

June 14, 2022

Last Update Submit

July 27, 2022

Conditions

Dermatitis, Atopic

Keywords

Eczema

Outcome Measures

Primary Outcomes (11)

Part A: Maximum Observed Serum Concentration (Cmax) of Functional Dupilumab
Serum concentration of functional dupilumab was reported.
Post-dose on Days 1, 3, 8, 18, and 29
Part A: Dose Normalized Maximum Observed Serum Concentration (Cmax/Dose) of Dupilumab
Dose normalized was calculated as Cmax obtained directly from the concentration versus time curve divided by dose. Cmax/dose was measured in Milligrams per Liter/Milligrams per Kilogram (\[mg/L\]/\[mg/kg\]).
Post-dose on Days 1, 3, 8, 18, and 29
Part A: Time to Reach the Maximum Serum Concentration (Tmax) of Dupilumab
Tmax was obtained directly from the concentration versus time curve.
Post-dose on Days 1, 3, 8, 18, and 29
Part A: Last Quantifiable Serum Concentration (Clast) of Dupilumab
Clast is the last measurable serum concentration of dupilumab.
Post-dose on Days 1, 3, 8, 18, and 29
Part A: Time of the Last Quantifiable Serum Concentration (Tlast) of Dupilumab
Tlast was defined as the last time point with a measurable serum concentration of dupilumab.
Post-dose on Days 1, 3, 8, 18, and 29
Part A: Area Under the Serum Concentration-Time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast) of Dupilumab
AUClast was defined as area under the serum concentration time-curve from zero to the last measured concentration.
Post-dose on Days 1, 3, 8, 18, and 29
Part A: Dose Normalized Area Under the Serum Concentration-Time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast/Dose) of Dupilumab
Dose normalized AUClast was calculated by AUClast/dose.
Post-dose on Days 1, 3, 8, 18, and 29
Part A: Number of Participants With at Least One Treatment-Emergent Adverse Event (TEAE)
Adverse Event (AE) was defined as any untoward medical occurrence in a participant administered a study drug which may/may not have a causal relationship with study drug. Serious AE (SAE) was defined as any untoward medical occurrence that resulted in any of following outcomes: death, life-threatening, required initial/prolonged in-participant hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect/considered as medically important event. TEAE was defined as AE starting/worsening after first intake of study drug. TEAEs included participants with both SAEs and non-SAEs. Number of participants with TEAEs is reported.
Baseline up to Week 4
Part A: Number of Participants With TEAEs by Severity According to Qualitative Toxicity Scale
Severity of TEAEs were graded using Qualitative Toxicity Scale, as follows: Mild: Participant is aware of the event or symptom, but the event or symptom is easily tolerated; Moderate: Participant experiences sufficient discomfort to interfere with or reduce his or her usual level of activity; Severe: Significant impairment of functioning: the participant is unable to carry out his or her usual activities. Number of participants with TEAEs by severity were reported.
Baseline up to Week 4
Part B: Percentage of Participants With Investigator's Global Assessment (IGA) Score 0 or 1 at Week 16
The IGA is an assessment scale used in clinical studies to rate the severity of AD globally, based on a 5-point scale ranging from 0 to 4 where 0 = clear; 1=almost clear; 2=mild; 3=moderate; 4=severe. A negative change from baseline indicated improvement. Percentage of participants with IGA score of '0' or '1' is reported.
Week 16
Part B: Percentage of Participants With Eczema Area and Severity Index (EASI) -75 (EASI-75) (≥75% Improvement From Baseline) at Week 16
The EASI score is used to measure the severity and extent of AD and measured erythema, infiltration, excoriation and lichenification on 4 anatomic regions of the body: head, trunk, upper, and lower extremities. The total EASI score ranges from 0 (minimum) to 72 (maximum) points, with the higher scores indicating the worse severity of AD. EASI-75 responders were the participants who achieved ≥75% overall improvement in EASI score from baseline at Week 16.
Week 16

Secondary Outcomes (26)

Part A: Number of Participants With Serious TEAEs and Severe TEAEs
Baseline up to Week 4
Part A: Percent Change From Baseline in EASI Score at Week 4
Week 4
Part A: Percent Change From Baseline in SCORing Atopic Dermatitis (SCORAD) Score at Week 4
Week 4
Part A: Percentage of Participants With IGA Score 0 or 1 at Week 4
Week 4
Part A: Number of Participants With at Least One Positive Treatment-Emergent Anti-Drug Antibodies (ADA)
Baseline up to Day 57
+21 more secondary outcomes

Study Arms (4)

Part A (Open label Dupilumab): Age cohorts 1 & 2

EXPERIMENTAL

Age cohort 1: ≥2 years old to \<6 years old Age cohort 2: ≥6 months to \<2 years old

Drug: Dupilumab

Part B (Double-blind): Dupilumab dose 1

EXPERIMENTAL

The results of part A will be used to guide the selection of dose levels and dosing frequency for part B.

Drug: Dupilumab

Part B (Double-blind): Dupilumab dose 2

EXPERIMENTAL

The results of part A will be used to guide the selection of dose levels and dosing frequency for part B.

Drug: Dupilumab

Part B (Double-Blind): Placebo

EXPERIMENTAL

Drug: Matching placebo

Interventions

DupilumabDRUG

Solution for injection, subcutaneous (SC)

Also known as: DUPIXENT®, REGN668, SAR231893

Part A (Open label Dupilumab): Age cohorts 1 & 2Part B (Double-blind): Dupilumab dose 1Part B (Double-blind): Dupilumab dose 2

Matching placeboDRUG

Solution for injection, subcutaneous (SC)

Part B (Double-Blind): Placebo

Eligibility Criteria

Age6 Months - 5 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

Diagnosis of atopic dermatitis (AD) according to the American Academy of Dermatology consensus criteria at the screening visit
Participants with documented recent history (within 6 months before the screening visit) of inadequate response to topical AD medication(s)
IGA score at screening and baseline visits
part A: IGA = 4
part B: IGA ≥3
EASI score at screening and baseline visits
part A: EASI ≥21
part B: EASI ≥16
Body Surface Area (BSA) involvement at screening and baseline visits
part A: ≥15%
part B: ≥10%
At least 11 (of a total of 14\*) applications of a topical emollient (moisturizer) during the 7 consecutive days immediately before the baseline visit (not including the day of randomization) (for part B of the study only)
Baseline worst scratch/itch score weekly average score for maximum scratch/itch intensity ≥4 (for part B of the study only)
At least 11 (of a total of 14) daily applications of low potency TCS during the 2-week TCS standardization period (beginning on day -14) leading up to the baseline visit (for part B of the study only).

You may not qualify if:

Prior treatment with dupilumab
History of important side effects of low potency topical corticosteroids (only applicable for part B of the study)
Having used immunosuppressive/immunomodulating drugs within 4 weeks before the baseline visit
Treatment with a live (attenuated) vaccine within 4 weeks before the baseline visit
Active chronic or acute infection requiring treatment with systemic antibiotics, antivirals, antiprotozoals, or antifungals within 2 weeks before the baseline visit.
Known or suspected immunodeficiency, known history of human immunodeficiency virus (HIV) infection or HIV seropositivity at the screening visit, established diagnosis of HBV infection or HBV seropositivity at screening, established diagnosis of HCV infection or HCV seropositivity at screening
History of malignancy at any time before the baseline visit
Diagnosed active endoparasitic infections or at high risk of these infections
Severe concomitant illness(es) that, in the investigator's judgment, would adversely affect the patient's participation in the study
Body weight \<5 kg or ≥30 kg at baseline (only applicable part B of the study)

Contact the study team to confirm eligibility.